Vor Biopharma Inc

Previous wording

Leveraging our expertise in HSC biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as hematopoietic cell transplants (“HCTs”) to patients. Once these cells engraft into bone marrow, the patient’s healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as VCAR33 ALLO, a chimeric antigen receptor (“CAR”)-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.

Current wording

Leveraging our expertise in HSC biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as hematopoietic cell transplants (“HCTs”) to patients. Once these cells engraft into bone marrow, the patient’s healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as antibody drug conjugates (“ADCs”) or VCAR33 ALLO, a chimeric antigen receptor (“CAR”)-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.

Previous wording

We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data for this trial most recently in December 2023. The data showed that primary neutrophil engraftment occurred in all eight patients treated with trem-cel. Three out of three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity typically seen with Mylotarg treatment. We expect to release additional engraftment and hematologic protection data in the second half of 2024. If successful, this trial will provide important validating evidence of the potential of trem-cel and our broader approach.

Current wording

We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data for this trial most recently in December 2023. The data showed that primary neutrophil engraftment occurred in all eight patients treated with trem-cel. Three out of three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity typically seen with Mylotarg treatment. We expect to release additional engraftment and hematologic protection data and additional Mylotarg pharmacokinetic analyses in the second half of 2024. If successful, this trial will provide important validating evidence of the potential of trem-cel and our broader approach.

Previous wording

VCAR33 ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33 ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33 ALLO in VBP301 and expect to treat multiple additional patients in the first half of 2024. We expect to report initial data in the second half of 2024.

Current wording

VCAR33 ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33 ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33 ALLO in VBP301 and have treated multiple additional patients through the first half of 2024. We expect to report initial data in the second half of 2024.

Previous wording

We have incurred significant operating losses since inception, including net losses of $30.8 million for the three months ended March 31, 2024 and $117.9 million for the year ended December 31, 2023. As of March 31, 2024, we had an accumulated deficit of $370.9 million.

Current wording

We have incurred significant operating losses since inception, including net losses of $27.8 million and $58.6 million, respectively, for the three and six months ended June 30, 2024, and $117.9 million for the year ended December 31, 2023. As of June 30, 2024, we had an accumulated deficit of $398.7 million.

Previous wording

As of March 31, 2024, we had cash, cash equivalents and marketable securities of $107.5 million. We expect that our cash, cash equivalents and marketable securities at March 31, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.

Current wording

As of June 30, 2024, we had cash, cash equivalents and marketable securities of $85.9 million. We expect that our cash, cash equivalents and marketable securities at June 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.

Previous wording

Comparison of Three Months Ended March 31, 2024 and 2023

Current wording

Comparison of Three and Six Months Ended June 30, 2024 and 2023

Previous wording

Research and development expenses were $24.3 million for the three months ended March 31, 2024, compared to $21.9 million for the three months ended March 31, 2023. The increase of $2.4 million was primarily attributable to an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities, an increase in manufacturing costs to support our trem-cel and VCAR33 ALLO programs, and an increase in clinical costs, partially offset by a decrease in preclinical research costs.

Current wording

Research and development expenses were $46.1 million for the six months ended June 30, 2024, compared to $45.8 million for the six months ended June 30, 2023. The increase of $0.3 million was primarily attributable to an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities and an increase in clinical trial costs to support our trem-cel and VCAR33 ALLO programs, offset in part by a decrease in preclinical expenses.

Previous wording

General and administrative expenses were $8.0 million for the three months ended March 31, 2024, compared to $8.5 million for the three months ended March 31, 2023. The decrease of $0.5 million was primarily due to a decrease in consulting and legal expenses, partially offset by an increase in personnel costs.

Current wording

General and administrative expenses were $7.2 million for the three months ended June 30, 2024, compared to $8.3 million for the three months ended June 30, 2023. The decrease of $1.1 million was primarily due to a decrease in consulting and legal expenses, partially offset by an increase in personnel costs.

Previous wording

Other income decreased by $0.5 million during the three months ended March 31, 2024, compared to the three months ended March 31, 2023. The decrease in other income was due to decreases in interest earned from our cash, cash equivalents and marketable securities.

Current wording

Other income decreased by $1.0 million during the three months ended June 30, 2024, compared to the three months ended June 30, 2023. Other income decreased by $1.5 million for the six months ended June 30, 2024, compared to the six months ended June 30, 2023. The decrease in other income in both periods was due to decreases in interest earned from our cash, cash equivalents and marketable securities as the Company held lower balances of cash, cash equivalents and marketable securities.

Previous wording

In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 139,462 shares of our common stock under the Stifel ATM Facility during the three months ended March 31, 2024 at a weighted-average price per share of $2.11 for aggregate net proceeds of $0.3 million, after deducting commissions. As of March 31, 2024, $119.8 million remained available to be sold under the Stifel ATM Facility.

Current wording

In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 139,462 shares of our common stock under the Stifel ATM Facility during the six months ended June 30, 2024 at a weighted-average price per share of $2.11 for aggregate net proceeds of $0.3 million, after deducting commissions. As of June 30, 2024, $119.8 million remained available to be sold under the Stifel ATM Facility.

Previous wording

As of March 31, 2024, we had cash, cash equivalents and marketable securities of $107.5 million. We will need to raise additional capital in the future to fund our future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us. In the event that we are unable to obtain sufficient additional funding, there can be no assurance that we will be able to continue as a going concern.

Current wording

As of June 30, 2024, we had cash, cash equivalents and marketable securities of $85.9 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.

Previous wording

We expect that our existing cash, cash equivalents and marketable securities at March 31, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

Current wording

Based on our current business plan and current capital resources, management has concluded that there is substantial doubt regarding our ability to continue as a going concern. We expect that our existing cash, cash equivalents and marketable securities at June 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

Previous wording

In addition, we expect to continue to incur additional costs associated with operating as a public company, including significant legal, audit, accounting, investor and public relations, regulatory, tax-related, director and officer insurance premiums, investor relations and other expenses. Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval for any product candidates or generate revenue from the sale of any product candidate for which we may obtain marketing approval. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for at least several years, if ever.

Current wording

Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval for any product candidates or generate revenue from the sale of any product candidate for which we may obtain marketing approval. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for at least several years, if ever.

Previous wording

Net cash used in operating activities was $24.3 million for the three months ended March 31, 2023, reflecting a net loss of $28.4 million and net cash used of $0.7 million for operating assets and liabilities, which were offset by non-cash charges of $4.8 million. The non-cash charges primarily consisted of stock-based compensation expense of $4.1 million, non-cash lease expense of $1.1 million and depreciation expense of $0.8 million, offset by $1.2 million of non-cash interest earned on marketable securities.

Current wording

Net cash used in operating activities was $52.3 million for the six months ended June 30, 2024, reflecting a net loss of $58.6 million and net cash used of $2.7 million for operating assets and liabilities, which were offset by non-cash charges of $9.0 million. The non-cash charges primarily consisted of stock-based compensation expense of $5.9 million, non-cash lease expense of $2.5 million and depreciation expense of $1.8 million, offset by $1.1 million of non-cash interest earned on marketable securities.

Previous wording

Net cash used in operating activities was $30.5 million for the three months ended March 31, 2024, reflecting a net loss of $30.8 million and net cash used of $4.1 million for operating assets and liabilities, which were offset by non-cash charges of $4.5 million. The non-cash charges primarily consisted of stock-based compensation expense of $3.1 million, non-cash lease expense of $1.2 million and depreciation expense of $0.9 million, offset by $0.8 million of non-cash interest earned on marketable securities.

Current wording

Net cash used in operating activities was $48.4 million for the six months ended June 30, 2023, reflecting a net loss of $58.4 million and net cash used of $0.2 million for operating assets and liabilities, which were offset by non-cash charges of $9.9 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.3 million, non-cash lease expense of $2.3 million and depreciation expense of $1.7 million, offset by $2.4 million of non-cash interest earned on marketable securities.

Previous wording

The $6.2 million increase in net cash used in operating activities for the three months ended March 31, 2024 compared to the three months ended March 31, 2023 was primarily due to an increase in research and development expenses as our clinical programs progressed and differences in the timing of payments for costs incurred during each respective period.

Current wording

The $3.9 million increase in net cash used in operating activities for the six months ended June 30, 2024 compared to the six months ended June 30, 2023 was primarily due to a decrease in stock-based compensation expense and differences in the timing of payments for costs incurred during each respective period.

Previous wording

Net cash provided by investing activities was $44.0 million for the three months ended March 31, 2024, which consisted of proceeds of $54.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.1 million of property and equipment. Net cash provided by investing activities was $6.5 million for the three months ended March 31, 2023, which consisted of proceeds of $30.0 million from the maturity of marketable securities, offset by purchases of $23.4 million of marketable securities and $0.1 million of property and equipment.

Current wording

Net cash provided by investing activities was $97.0 million for the six months ended June 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.1 million of property and equipment. Net cash provided by investing activities was $20.1 million for the six months ended June 30, 2023, which consisted of purchases of $58.4 million of marketable securities and $0.5 million of property and equipment offset by proceeds of $79.0 million from the maturity of marketable securities.

Previous wording

Net cash provided by financing activities was $0.1 million for the three months ended March 31, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million, offset by $0.2 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.5 million for the three months ended March 31, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $3.6 million, offset by $0.4 million of taxes paid related to net share settlement of equity awards and $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022.

Current wording

Net cash provided by financing activities was $0.1 million for the six months ended June 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.2 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.2 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.7 million for the six months ended June 30, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $4.2 million, and proceeds from stock option exercises and purchases of common stock under the ESPP of $0.2 million offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $0.9 million of taxes paid related to net share settlement of equity awards.

Previous wording

Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the three months ended March 31, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2023 Annual Report.

Current wording

Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the six months ended June 30, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2023 Annual Report.